E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pharmacokinetics in subjects with PID |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To compare the AUC0-7 days of IgG during the last week of each study period (Weeks 4 & 8) after subcutaneous (SC) infusion of the same IgPro20 dose with the IWI vs CP in PID patients |
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E.2.2 | Secondary objectives of the trial |
Compare pharmacokinetic (PK) parameters and the safety and local tolerability of IgG after subcutaneous (SC) infusion of the same IgPro20 dose with the IWI vs CP in PID patients. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female.
2. At least 12 years of age at the time of providing written informed consent/assent.
3. Diagnosis of PID |
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E.4 | Principal exclusion criteria |
1. Newly diagnosed PID; ie, subjects who have not previously received SCIG replacement therapy.
2. Ongoing active serious infection at the time of screening
3. Ongoing or history of concomitant malignancies of lymphoid cells, such as lymphocytic leukemia, non-Hodgkin’s lymphoma, or immunodeficiency with lymphoma.
4. Known hyperprolinemia. |
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to 7 days after infusion of the last week of each study period (Weeks 4 and 8) |
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E.5.2 | Secondary end point(s) |
- Serum IgG Cmax
- Serum IgG Ctrough
- Serum IgG Ctrough
- Number and percentage of subjects with any adverse events (AEs)
- Number and percentage of subjects with AEs attributed to IgPro20 only
- Number and percentage of subjects with AEs attributed to each device (IWI and CP)
- Number and percentage of subjects with AEs attributed to drug/device combination (IgPro_20 / IWI and IgPro_20 / CP)
- Number and percentage of subjects with serious AEs
- Time to onset of injection site reactions
- Duration of injection site reactions |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Measured during a 1-week PK collection period
- Measured during a 1-week PK collection period
- Before every weekly infusion
- Up to 8 weeks
- Up to 8 weeks
- Up to 4 weeks
- Up to 4 weeks
- Up to 8 weeks
- Up to 7 days after infusion
- Up to 8 weeks
- Up to 8 weeks |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Comparison of two infusion devices |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Device. PK from IgPro20 to be measured and compared between the two devices: IWI vs CP |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 2 |